SYDNEY, April 25, 2016 /PRNewswire/ -- Benitec
Biopharma Limited (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW), a
biotechnology company developing innovative therapeutics based on
its patented gene-silencing technology called ddRNAi or 'expressed
RNAi', today announced acceptance of five presentations at the
19th Annual Meeting of American Society of Gene and Cell
Therapy (ASGCT) being held in Washington
D.C. on May 4-7, 2016.
The abstracts cover the scientific results from three of Benitec's
programs: hepatitis B, OPMD and hepatitis C, with the key findings
briefly summarised below.
David Suhy, Chief Scientific
Officer of Benitec Biopharma, stated: "This meeting is one of the
most important international meetings on gene and cell therapy. To
have five abstracts accepted demonstrates the interest and
importance in the work that Benitec is doing across its programs
and will give our research further exposure to a global audience.
As we move ahead with our programs, we will update both the
scientific and investor communities on our clinical priorities and
next phases of development."
1. Oral Presentation:
BB-HB-331, a DNA-Directed RNA Interference (ddRNAi) Agent
Targeting Hepatitis B Virus (HBV), Can Effectively Suppress HBV
In Vitro and In Vivo
Presenter: David Suhy, PhD
- In vitro treatment of PXB-derived primary human
hepatocytes with Ad-BB-HB-331 led to significant decreases in HBV
parameters with dose dependent expression of the anti-HBV shRNAs
and corresponding inhibition of the HBV viral RNAs.
- Similarly, effective suppression of HBV infection was observed
following in vivo treatment of PXB mice with
AAV8-BB-HB-331.
- Through the first 28 days of an ongoing 56-day experiment,
treatment with the high dose resulted in decreases in extracellular
levels of HBsAg and HBeAg by 90% and 84%, respectively, when
compared to the untreated control. In addition, treatment with the
same dose resulted in nearly a log reduction of extracellular HBV
DNA at 28 days.
In a press release dated
March 8, 2016, Benitec reported the
data from the full 56-day in vivo study which demonstrated a
further marked improvement over the results above. Dr Suhy
will present the full data set at the conference.
2. Oral Presentation:
Gene Therapy Rescues Disease Phenotype in the Oculopharyngeal
Muscular Dystrophy Mouse Model
Presenter: David Suhy, PhD (on
behalf of Professor George
Dickson)
- Treating mice affected by OPMD over 4 months with an AAV gene
therapy strategy based on DNA-directed RNA interference to silence
the endogenous expPABPN1, combined with the re-expression of a
healthy sequence-optimized human PABPN1 gene, significantly reduces
the amount of nuclear aggregates in affected muscles, decreased the
intramuscular fibrosis and reverted the muscle strength to the
level of healthy wild-type muscles.
- Although muscle atrophy was not reverted, the expression of a
healthy PABPN1 markedly increased the cross sectional area of
muscle fibres.
3. Oral Presentation: Phase
I/IIa Study of TT-034, a DNA-Directed RNA Interference (ddRNAi)
Agent Delivered as a Single Administration for the Treatment of
Subjects with Chronic Hepatitis C Virus (HCV)
Presenter:
David Suhy, PhD
- The three doses of TT-034 administered to date have been well
tolerated in human subjects infected with the hepatitis C virus
(HCV) and there have been no reported serious adverse events
related to administration of the study drug.
- The initial dose (4E10 vg/kg) resulted in very low levels of
transduction as expected.
- The second dose (1.25E11 vg/kg) resulted in the detection of
substantially higher levels of TT-034 in hepatocytes, the
predominant cell type in the liver, yielding 0.48, 3.65 and 10.44
copies of TT-034 DNA per cell in the three patients,
respectively.
- The first subject administered with the third dose (4.00E11
vg/kg) had 17.74 copies of TT-034 per cell, indicating that a
significant portion of their hepatocytes may have been transduced,
and expression of anti-HCV shRNAs was clearly detected in the
transduced hepatocytes.
4. Poster: Durable Expression
of TT-034 in Cynomolgus Monkey Hepatic and Cardiac Tissues without
Long-Term Adverse Effects on Endogenous MicroRNA
Levels
Presenter: David Suhy,
PhD
- A single, intravenous infusion of TT-034 results in almost
complete transduction of Cynomolgus monkey hepatocytes and results
in durable expression of three anti-HCV shRNAs.
- The doses of TT-034, meant to encompass the range of doses
administered in a human clinical study, do not cause long-term
perturbation of endogenous miRNA levels.
5. Poster: A Comparison of
scAAV8-TT-034 Mediated Transduction and shRNA Expression in Human
Liver Biopsy Samples versus a Chimeric Mouse Model with Humanized
Liver
Presenter: David Suhy,
PhD
- Residual mouse hepatocytes present in the chimeric livers are
transduced more efficiently with the scAAV8 vector than human
hepatocytes resulting in lower overall transduction and shRNA
expression as compared to similar data obtained from the TT-034
human clinical samples.
- While these models can serve as a surrogate to assess the
activity of gene therapy constructs against functions of normal
human liver, the doses required for optimal activity may be
modestly higher than required in the human clinical setting.
The full abstracts have been
published on the conference website and can be accessed at the
following link: http://www.abstractsonline.com/pp8/#!/4077. A
copy of the oral presentations and posters will be accessible on
May 4, 2016 by visiting the Investor
sections of www.benitec.com and selecting News/Resources and
Presentations.
For further information regarding Benitec and its activities,
please contact the persons below, or visit the Benitec website at
www.benitec.com
Company
|
Investor
relations
|
United States
|
|
|
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Carl Stubbings
|
Buchan Consulting
|
PCG Advisory Group
|
Chief Business Officer
|
Kyahn Williamson
|
Adam Holdsworth
|
Tel: +61 (2) 9555 6986
|
Head of Investor Communications
|
Managing Director of Investor
Relations
|
Email: cstubbings@benitec.com
|
Tel: +61 (3) 9866 4722
|
Tel: + 1 646-862-4607
|
|
Email:
kwilliamson@buchanwe.com.au
|
Email: adamh@pcgadvisory.com
|
|
|
|
|
|
Sean Leous
|
|
|
Managing Director of Public
Relations
|
|
|
Tel: +1 646-863-8998
|
|
|
Email: sleous@pcgadvisory.com
|
About Benitec Biopharma Limited:
Benitec
Biopharma Limited (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) is a
biotechnology company developing innovative therapeutics based on
its patented gene-silencing technology called ddRNAi or 'expressed
RNAi'. Based in Sydney, Australia
with labs in Hayward, CA (USA) and
collaborators and licensees around the world, the company is
developing ddRNAi-based therapeutics for chronic and
life-threatening human conditions including hepatitis B, wet
age-related macular degeneration and OPMD. Benitec has also
licensed ddRNAi to other biopharmaceutical companies for
applications including HIV/AIDS, Huntington's Disease, chronic
neuropathic pain and retinitis pigmentosa.
Safe Harbor Statement:
This press release
contains "forward-looking statements" within the meaning of section
27A of the US Securities Act of 1933 and section 21E of the US
Securities Exchange Act of 1934. Any forward-looking statements
that may be in the press release are subject to risks and
uncertainties relating to the difficulties in Benitec's plans to
develop and commercialize its product candidates, the timing of the
initiation and completion of preclinical and clinical trials, the
timing of patient enrolment and dosing in clinical trials, the
timing of expected regulatory filings, the clinical utility and
potential attributes and benefits of ddRNAi and Benitec's product
candidates, potential future out-licenses and collaborations, the
intellectual property position and the ability to procure
additional sources of financing. Accordingly, you should not rely
on those forward-looking statements as a prediction of actual
future results.
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visit:http://www.prnewswire.com/news-releases/benitec-to-present-at-the-asgct-2016-annual-meeting-300256558.html
SOURCE Benitec Biopharma Limited